IL220219A0 - Rna preparations comprising purified modified rna for reprogramming cells - Google Patents

Rna preparations comprising purified modified rna for reprogramming cells

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Publication number
IL220219A0
IL220219A0 IL220219A IL22021912A IL220219A0 IL 220219 A0 IL220219 A0 IL 220219A0 IL 220219 A IL220219 A IL 220219A IL 22021912 A IL22021912 A IL 22021912A IL 220219 A0 IL220219 A0 IL 220219A0
Authority
IL
Israel
Prior art keywords
rna
purified modified
preparations
reprogramming cells
modified rna
Prior art date
Application number
IL220219A
Other languages
English (en)
Original Assignee
Katalin Kariko
Drew Weissman
Judith Meis
Gary Dahl
Jerome Jendrisak
Anthony Person
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Katalin Kariko, Drew Weissman, Judith Meis, Gary Dahl, Jerome Jendrisak, Anthony Person filed Critical Katalin Kariko
Publication of IL220219A0 publication Critical patent/IL220219A0/en

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Families Citing this family (151)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
HUE043492T2 (hu) 2005-08-23 2019-08-28 Univ Pennsylvania Módosított nukleozidokat tartalmazó RNS és eljárások az alkalmazására
US8859229B2 (en) * 2007-02-02 2014-10-14 Yale University Transient transfection with RNA
US9249423B2 (en) * 2007-02-02 2016-02-02 Yale University Method of de-differentiating and re-differentiating somatic cells using RNA
US10155038B2 (en) 2007-02-02 2018-12-18 Yale University Cells prepared by transient transfection and methods of use thereof
DK3338765T3 (en) 2009-12-01 2019-03-04 Translate Bio Inc STEROID DERIVATIVE FOR THE SUPPLY OF MRNA IN HUMANGENETIC DISEASES
LT3112467T (lt) 2009-12-07 2018-06-25 The Trustees Of The University Of Pennsylvania Išgrynintą modifikuotą rnr apimantys rnr preparatai, skirti ląstelių perprogramavimui
CA2796464C (en) 2010-04-16 2021-08-03 Immune Disease Institute, Inc. Sustained polypeptide expression from synthetic, modified rnas and uses thereof
ES2557382T3 (es) 2010-07-06 2016-01-25 Glaxosmithkline Biologicals Sa Liposomas con lípidos que tienen un valor de pKa ventajoso para el suministro de ARN
PL3243526T3 (pl) 2010-07-06 2020-05-18 Glaxosmithkline Biologicals S.A. Dostarczanie rna w celu wyzwolenia wielu szlaków immunologicznych
PL2591114T3 (pl) 2010-07-06 2017-08-31 Glaxosmithkline Biologicals Sa Immunizacja dużych ssaków małymi dawkami rna
EP3578205A1 (en) 2010-08-06 2019-12-11 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
HUE061068T2 (hu) 2010-08-31 2023-05-28 Glaxosmithkline Biologicals Sa Pegilált liposzómák immunogént kódoló RNS szállítására
CN103429606A (zh) 2010-10-01 2013-12-04 现代治疗公司 设计核酸及其使用方法
EP4098324A1 (en) 2010-10-11 2022-12-07 GlaxoSmithKline Biologicals S.A. Antigen delivery platforms
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
JP6184945B2 (ja) 2011-06-08 2017-08-23 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド mRNA送達のための脂質ナノ粒子組成物および方法
CA2840989A1 (en) 2011-07-06 2013-01-10 Novartis Ag Immunogenic combination compositions and uses thereof
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
KR20190099538A (ko) * 2011-10-03 2019-08-27 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드, 및 핵산, 및 이들의 용도
BR112014013664A2 (pt) 2011-12-05 2020-11-03 Factor Bioscience Inc. métodos e produtos para transfectar células
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
US20140343129A1 (en) * 2011-12-14 2014-11-20 Moderna Therapeutics, Inc. Modified nucleic acids, and acute care uses thereof
EP4144378A1 (en) 2011-12-16 2023-03-08 ModernaTX, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
CA2859691A1 (en) * 2011-12-21 2013-06-27 Moderna Therapeutics, Inc. Methods of increasing the viability or longevity of an organ or organ explant
LT3144389T (lt) * 2011-12-30 2018-08-10 Cellscript, Llc In vitro susintetintos viengrandės rnr gavimas ir panaudojimas įvedimui į žinduolio ląsteles, siekiant sužadinti biologinį arba biocheminį poveikį
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
AU2013243954A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
AU2013243949A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
JP6448531B2 (ja) * 2012-05-13 2019-01-09 アリール バイオテクノロジー アンド ファーマシューティカルズ, インコーポレイテッド 合成メッセンジャーrnaを使用したヒト人工多能性幹細胞のフィーダーフリー誘導
US10245229B2 (en) * 2012-06-08 2019-04-02 Translate Bio, Inc. Pulmonary delivery of mRNA to non-lung target cells
EP3536787A1 (en) 2012-06-08 2019-09-11 Translate Bio, Inc. Nuclease resistant polynucleotides and uses thereof
US9512456B2 (en) 2012-08-14 2016-12-06 Modernatx, Inc. Enzymes and polymerases for the synthesis of RNA
CA2883714A1 (en) 2012-09-07 2014-03-13 Genentech, Inc. Methods and compositions for producing induced hepatocytes
WO2014062801A1 (en) 2012-10-16 2014-04-24 Massachusetts Institute Of Technology Production of stable non-polyadenylated rnas
BR122019025681B1 (pt) * 2012-11-01 2023-04-18 Factor Bioscience Inc Método para inserir uma sequência de ácido nucleico em uma localização segura de um genoma de uma célula
DK2922554T3 (en) 2012-11-26 2022-05-23 Modernatx Inc Terminalt modificeret rna
EP4331620A3 (en) * 2012-12-07 2024-12-04 Translate Bio, Inc. Lipidic nanoparticles for mrna delivery
PT2959005T (pt) 2013-02-22 2021-12-30 Univ Leland Stanford Junior Utilização médica relacionada com extensão de telómero
EP2968391A1 (en) 2013-03-13 2016-01-20 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
JP6586075B2 (ja) 2013-03-14 2019-10-02 トランスレイト バイオ, インコーポレイテッド メッセンジャーrnaの精製方法
IL305374A (en) 2013-03-14 2023-10-01 Ethris Gmbh Cftr mrna compositions and related methods and uses
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
WO2014160243A1 (en) * 2013-03-14 2014-10-02 The Trustees Of The University Of Pennsylvania Purification and purity assessment of rna molecules synthesized with modified nucleosides
WO2014152030A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Removal of dna fragments in mrna production process
WO2014152027A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Manufacturing methods for production of rna transcripts
EP3578652B1 (en) 2013-03-15 2023-07-12 ModernaTX, Inc. Ribonucleic acid purification
US8980864B2 (en) * 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
EP2983804A4 (en) 2013-03-15 2017-03-01 Moderna Therapeutics, Inc. Ion exchange purification of mrna
EP2996697B1 (en) 2013-05-15 2019-06-26 Robert Kruse Intracellular translation of circular rna
DK3019619T3 (da) 2013-07-11 2021-10-11 Modernatx Inc Sammensætninger, der omfatter syntetiske polynukleotider, som koder for crispr-beslægtede proteiner, og syntetiske sgrna'er, og anvendelsesfremgangsmåder
JP6516672B2 (ja) * 2013-07-26 2019-05-22 京都府公立大学法人 骨芽細胞及びその調製方法
ES2857908T3 (es) * 2013-08-28 2021-09-29 Becton Dickinson Co Análisis masivamente paralelo de células individuales
AU2014315287A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
US10385088B2 (en) 2013-10-02 2019-08-20 Modernatx, Inc. Polynucleotide molecules and uses thereof
BR112016007255A2 (pt) 2013-10-03 2017-09-12 Moderna Therapeutics Inc polinucleotídeos que codificam receptor de lipoproteína de baixa densidade
BR112016009014B1 (pt) 2013-10-22 2024-02-06 Translate Bio, Inc USO DE COMPOSIÇÃO COMPREENDENDO mRNA PARA DEFICIÊNCIA DE ARGININOSSUCINATO SINTETASE
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
EP3062798B1 (en) 2013-11-01 2020-05-06 CureVac AG Modified rna with decreased immunostimulatory properties
CA2925021C (en) 2013-11-01 2025-05-06 Curevac Ag MODIFIED MESSENGER RIBONUCLE ACID (MRNA) WITH REDUCED IMMUNOSTIMULATING PROPERTIES
KR101665165B1 (ko) * 2013-12-04 2016-10-12 단국대학교 산학협력단 신규한 rna 앱타머 및 그의 용도
US9770489B2 (en) 2014-01-31 2017-09-26 Factor Bioscience Inc. Methods and products for nucleic acid production and delivery
WO2015120225A1 (en) 2014-02-10 2015-08-13 The Board Of Trustees Of The Leland Stanford Junior University ACTIVATION OF INNATE IMMUNITY FOR ENHANCED NUCLEAR REPROGRAMMING OF SOMATIC CELLS WITH mRNA
PT3981437T (pt) 2014-04-23 2025-01-15 Modernatx Inc Vacinas de ácidos nucleicos
MX373952B (es) 2014-04-25 2020-07-13 Shire Human Genetic Therapies Métodos de purificación de arn mensajero.
US10286086B2 (en) 2014-06-19 2019-05-14 Modernatx, Inc. Alternative nucleic acid molecules and uses thereof
EP3169335B8 (en) 2014-07-16 2019-10-09 ModernaTX, Inc. Circular polynucleotides
WO2016011226A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
EP3041948B1 (en) 2014-11-10 2019-01-09 Modernatx, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
US11241505B2 (en) 2015-02-13 2022-02-08 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
DE112016002962T5 (de) 2015-06-30 2018-05-24 Ethris Gmbh Familie der ATP-bindenden Kassetten-codierende Polyribonucleotide und Formulierungen derselben
WO2017044853A1 (en) * 2015-09-09 2017-03-16 Trustees Of Tufts College Methods of generating neural stem cells
WO2017049275A2 (en) 2015-09-17 2017-03-23 Moderna Therapeutics, Inc. Polynucleotides containing a stabilizing tail region
US11434486B2 (en) 2015-09-17 2022-09-06 Modernatx, Inc. Polynucleotides containing a morpholino linker
WO2017051347A2 (en) * 2015-09-23 2017-03-30 Pfizer Inc. Cells and method of cell culture
JP6990176B2 (ja) 2015-10-05 2022-02-03 モデルナティエックス インコーポレイテッド メッセンジャーリボ核酸薬物の治療投与のための方法
WO2017180587A2 (en) 2016-04-11 2017-10-19 Obsidian Therapeutics, Inc. Regulated biocircuit systems
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
ES2900272T3 (es) 2016-04-22 2022-03-16 BioNTech SE Métodos para proporcionar ARN monocatenario
IL263079B2 (en) 2016-05-18 2024-05-01 Modernatx Inc Polynucleotides encoding relaxin
US20190224335A1 (en) * 2016-06-03 2019-07-25 Stemgenics, Inc. Direct reprogramming of a human somatic cell to a selected (predetermined) differentiated cell with functionalized nanoparticles
WO2017223176A1 (en) 2016-06-24 2017-12-28 Modernatx, Inc. Methods and apparatus for filtration
CN106226524B (zh) * 2016-07-07 2018-10-09 福建省农业科学院食用菌研究所 一种食用菌dsRNA病毒的检测方法
US10576167B2 (en) 2016-08-17 2020-03-03 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
CA3036831A1 (en) 2016-09-14 2018-03-22 Modernatx, Inc. High purity rna compositions and methods for preparation thereof
ES2967788T3 (es) * 2016-09-16 2024-05-03 Icahn School Med Mount Sinai Expresión especifica de célula de Arnmond
EP3538146A4 (en) 2016-11-11 2020-07-15 ModernaTX, Inc. INFLUENZA VACCINE
EP3585417B1 (en) 2017-02-27 2023-02-22 Translate Bio, Inc. Method of making a codon-optimized cftr mrna
UY37621A (es) 2017-02-28 2018-09-28 Sanofi Sa Arn terapéutico que codifica citoquinas
WO2018188730A1 (en) 2017-04-11 2018-10-18 Biontech Rna Pharmaceuticals Gmbh Rna for treatment of autoimmune diseases
CN116555353A (zh) 2017-04-20 2023-08-08 E开创生物技术股份有限公司 产生基因修改的动物的方法
EP3624824B1 (en) 2017-05-16 2024-07-10 Translate Bio, Inc. Codon-optimized mrna encoding cftr for use in treating cystic fibrosis
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS
US10034951B1 (en) 2017-06-21 2018-07-31 New England Biolabs, Inc. Use of thermostable RNA polymerases to produce RNAs having reduced immunogenicity
MA49914A (fr) 2017-08-18 2021-04-21 Modernatx Inc Procédés analytiques par hplc
US11912982B2 (en) 2017-08-18 2024-02-27 Modernatx, Inc. Methods for HPLC analysis
CN107563149B (zh) * 2017-08-21 2020-10-23 上海派森诺生物科技股份有限公司 全长转录本的结构注释和比对结果评估方法
CN111315359A (zh) 2017-08-31 2020-06-19 摩登纳特斯有限公司 制备脂质纳米颗粒的方法
CA3075696A1 (en) * 2017-09-13 2019-03-21 Biontech Rna Pharmaceuticals Gmbh Method of enhancing rna expression in a cell
CR20200462A (es) * 2018-03-13 2020-12-03 Univ Leland Stanford Junior Reprogramación celular trasitoria para revertir el envejecimiento celular
WO2019241315A1 (en) 2018-06-12 2019-12-19 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
CN108676855B (zh) * 2018-07-24 2021-09-28 内蒙古赛科星家畜种业与繁育生物技术研究院有限公司 标准曲线法荧光定量pcr鉴定牛转基因拷贝数的方法及引物
CA3108544A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
US11072808B2 (en) 2018-10-04 2021-07-27 New England Biolabs, Inc. Methods and compositions for increasing capping efficiency of transcribed RNA
CA3114892A1 (en) 2018-10-04 2020-04-09 New England Biolabs, Inc. Methods and compositions for increasing capping efficiency of transcribed rna
US20210386788A1 (en) 2018-10-24 2021-12-16 Obsidian Therapeutics, Inc. Er tunable protein regulation
US12174174B2 (en) * 2018-10-31 2024-12-24 National Cancer Center Composition comprising material for regulating OCT4 modification to repress stemness
AU2019384557B2 (en) 2018-11-21 2025-07-17 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
WO2020168466A1 (en) * 2019-02-19 2020-08-27 Stemirna Therapeutics Co., Ltd. Modified nucleoside and synthetic methods thereof
CN113874502A (zh) 2019-03-11 2021-12-31 摩登纳特斯有限公司 补料分批体外转录方法
KR20220039719A (ko) 2019-07-03 2022-03-29 팩터 바이오사이언스 인크. 양이온성 지질 및 이의 용도
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
CN112390838A (zh) * 2019-08-14 2021-02-23 斯微(上海)生物科技有限公司 一种改性核苷及其合成方法
WO2021030533A1 (en) * 2019-08-14 2021-02-18 Modernatx, Inc. Processes for purifying downstream products of in vitro transcription
CN110894493A (zh) * 2019-10-28 2020-03-20 吉林大学 一种重编程间充质干细胞及其制备方法
AU2020383433A1 (en) * 2019-11-11 2022-06-23 Novoxyne LLC Method and a kit to reprogram somatic cells
WO2021149824A1 (ja) * 2020-01-24 2021-07-29 アイ ピース, インコーポレイテッド 人工多能性幹細胞の作製方法
US12194089B2 (en) 2020-02-04 2025-01-14 CureVac SE Coronavirus vaccine
KR20210116324A (ko) 2020-03-12 2021-09-27 기초과학연구원 게놈 서열 변이를 가지는 세포 사멸 유도용 조성물 및 상기 조성물을 이용한 세포 사멸 유도 방법
US12194157B2 (en) 2020-04-09 2025-01-14 Finncure Oy Carrier for targeted delivery to a host
AU2021252164A1 (en) 2020-04-09 2022-12-15 Finncure Oy Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses
KR20230173074A (ko) * 2020-11-13 2023-12-26 이제네시스, 인크. 향상된 이종이식편 생존 및 관용을 위한 하나 이상의 변형된 유전자를 갖는 세포, 조직, 기관, 및 동물
AU2021405281A1 (en) 2020-12-22 2023-07-06 Glaxosmithkline Biologicals Sa Rna vaccine against sars-cov-2 variants
JP2022104813A (ja) 2020-12-29 2022-07-11 アイ ピース,インコーポレイテッド リプログラミング因子を導入された細胞の培養方法
US12329811B2 (en) 2021-01-11 2025-06-17 Modernatx, Inc. Seasonal RNA influenza virus vaccines
CN113408945B (zh) * 2021-07-15 2023-03-24 广西中烟工业有限责任公司 一种烤烟纯度的检测方法、装置、电子设备及存储介质
WO2023288288A1 (en) 2021-07-15 2023-01-19 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs with on/off mechanism for controlled expression and methods of use
WO2023288285A1 (en) * 2021-07-15 2023-01-19 Turn Biotechnologies, Inc. Polycistronic expression vectors
US20230044997A1 (en) 2021-07-15 2023-02-09 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs and methods of use for cell rejuvenation and for treatment
CN114196676B (zh) * 2021-11-29 2023-07-18 中国农业科学院北京畜牧兽医研究所 Itga2基因在调控猪米色脂肪形成中的应用
CN114561381A (zh) * 2022-03-14 2022-05-31 桂林医学院 免疫mRNA及其制备方法和应用
CN114540387B (zh) * 2022-03-28 2024-04-26 仁景(苏州)生物科技有限公司 一种ires序列介导的非帽依赖型的基因表达载体及其应用
KR102785258B1 (ko) * 2022-09-07 2025-03-26 아이진 주식회사 변형핵산 함유 mRNA의 체내 전달용 조성물
WO2024054020A1 (ko) 2022-09-07 2024-03-14 아이진 주식회사 변형핵산 함유 mrna의 체내 전달용 조성물
WO2024089638A1 (en) 2022-10-28 2024-05-02 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine
CN115992146B (zh) * 2022-11-11 2025-04-18 上海交通大学医学院附属第九人民医院 经修饰的mRNA分子及相关产品和用途
EP4633646A2 (en) * 2022-12-14 2025-10-22 Grann Pharmaceuticals Inc. Compositions and methods for modulating hsp70 activity
WO2024249954A1 (en) 2023-05-31 2024-12-05 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025024442A1 (en) * 2023-07-26 2025-01-30 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating conditions associated with bsep deficiency
CN116898988A (zh) * 2023-08-15 2023-10-20 臻赫医药(杭州)有限公司 皮肤重编程因子制剂、生物材料及用途
WO2025038982A2 (en) * 2023-08-17 2025-02-20 Duke University Regulators of t cell exhaustion
WO2025144938A1 (en) 2023-12-26 2025-07-03 Emmune, Inc. Systems for nucleic acid transfer
CN117511947B (zh) * 2024-01-08 2024-03-29 艾斯拓康医药科技(北京)有限公司 一种优化的5`utr序列及其应用
KR20250110708A (ko) 2024-01-11 2025-07-21 서울대학교산학협력단 역분화 유전자의 간 특이적 전달을 통한 간 재생 방법
CN119307519B (zh) * 2024-07-22 2025-11-25 苏州剂泰医药科技有限公司 编码荧光素酶的mRNA及其应用

Family Cites Families (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5824497A (en) * 1995-02-10 1998-10-20 Mcmaster University High efficiency translation of mRNA molecules
JP2002507121A (ja) * 1997-06-25 2002-03-05 プロメガ コーポレイション Rnaの単離方法
AU9319398A (en) * 1997-09-19 1999-04-05 Sequitur, Inc. Sense mrna therapy
CA2437737A1 (en) 2001-02-14 2002-08-22 Stephen D. Ginsberg Methods and compositions of amplifying rna
FR2822164B1 (fr) 2001-03-19 2004-06-18 Centre Nat Rech Scient Polypeptides derives des arn polymerases, et leurs utilisations
US20050137155A1 (en) 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
US8137911B2 (en) 2001-05-22 2012-03-20 Cellscript, Inc. Preparation and use of single-stranded transcription substrates for synthesis of transcription products corresponding to target sequences
WO2004048594A2 (en) 2002-11-21 2004-06-10 Epicentre Technologies Preparation and use of single-stranded transcription substrates for synthesis of transcription products corresponding to target sequences
US9567591B2 (en) * 2003-05-15 2017-02-14 Mello Biotechnology, Inc. Generation of human embryonic stem-like cells using intronic RNA
US20050153333A1 (en) 2003-12-02 2005-07-14 Sooknanan Roy R. Selective terminal tagging of nucleic acids
EP1773857A4 (en) 2004-07-02 2009-05-13 Protiva Biotherapeutics Inc THE IMMUNE SYSTEM STIMULATING SIRNA MOLECULES AND APPLICATIONS THEREOF
HUE043492T2 (hu) 2005-08-23 2019-08-28 Univ Pennsylvania Módosított nukleozidokat tartalmazó RNS és eljárások az alkalmazására
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
US20070087437A1 (en) * 2005-10-14 2007-04-19 Jifan Hu Methods for rejuvenating cells in vitro and in vivo
WO2007120863A2 (en) 2006-04-14 2007-10-25 Epicentre Technologies Kits and methods for generating 5' capped rna
DE102006051516A1 (de) * 2006-10-31 2008-05-08 Curevac Gmbh (Basen-)modifizierte RNA zur Expressionssteigerung eines Proteins
KR101107256B1 (ko) 2007-03-27 2012-01-19 삼성전자주식회사 모션 벡터에 기반한 적응적 프레임율 변환 방법 및 장치 및적응적 프레임율 변환 기능을 가지는 디스플레이 장치
WO2008151058A2 (en) 2007-05-30 2008-12-11 The General Hospital Corporation Methods of generating pluripotent cells from somatic cells
US8039214B2 (en) 2007-06-29 2011-10-18 Cellscript, Inc. Synthesis of tagged nucleic acids
EP2072618A1 (en) 2007-12-14 2009-06-24 Johannes Gutenberg-Universität Mainz Use of RNA for reprogramming somatic cells
GB0801215D0 (en) * 2008-01-23 2008-02-27 Univ Sheffield Cell re-programming
WO2009101407A2 (en) * 2008-02-11 2009-08-20 Cambridge Enterprise Limited Improved reprogramming of mammalian cells, and the cells obtained
WO2009127230A1 (en) * 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
US8669048B2 (en) * 2008-06-24 2014-03-11 Parkinson's Institute Pluripotent cell lines and methods of use thereof
GB0905507D0 (en) 2009-03-31 2009-05-13 Dow Corning Organopol Ysiloxane Compositions Containing An Active Material
WO2010123501A1 (en) 2009-04-22 2010-10-28 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
ES2587963T3 (es) 2009-07-31 2016-10-27 Ethris Gmbh ARN con una combinación de nucleótidos no modificados y modificados para la expresión de proteínas
LT3112467T (lt) 2009-12-07 2018-06-25 The Trustees Of The University Of Pennsylvania Išgrynintą modifikuotą rnr apimantys rnr preparatai, skirti ląstelių perprogramavimui
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
CA2796464C (en) 2010-04-16 2021-08-03 Immune Disease Institute, Inc. Sustained polypeptide expression from synthetic, modified rnas and uses thereof
EP3578205A1 (en) 2010-08-06 2019-12-11 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
CN103429606A (zh) 2010-10-01 2013-12-04 现代治疗公司 设计核酸及其使用方法
WO2012148586A1 (en) 2011-03-15 2012-11-01 The Board Of Trustees Of The Leland Stanford Junior University Gpcr fusion protein containing an n-terminal autonomously folding stable domain, and crystals of the same
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US9862926B2 (en) 2011-06-27 2018-01-09 Cellscript, Llc. Inhibition of innate immune response
LT3144389T (lt) 2011-12-30 2018-08-10 Cellscript, Llc In vitro susintetintos viengrandės rnr gavimas ir panaudojimas įvedimui į žinduolio ląsteles, siekiant sužadinti biologinį arba biocheminį poveikį

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